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Volanesorsen may reduce burden of familial chylomicronemia syndrome
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Volanesorsen appears to reduce the burden of familial chylomicronemia syndrome, according to the results of a retrospective survey.
Volanesorsen (Akcea Therapeutics) is under review by the FDA and if approved would be the first available drug treatment for familial chylomicronemia syndrome (FCS), a rare disease caused by impaired function of lipoprotein lipase in which patients have severe hypertriglyceridemia and are at high risk for acute pancreatitis, which is sometimes fatal.
For the ReFOCUS study, 22 patients with FCS (median age, 51 years; 73% women) who received volanesorsen for at least 3 months in an open-label extension study took a web-based survey about their experiences before and after taking the drug to characterize the effect of volanesorsen on disease burden.
The percentage of patients who said their strategies for managing their disease were effective rose from 19% at baseline to 40% after treatment with volanesorsen, Marcello Arca, MD, from La Sapienza University of Rome, and colleagues wrote.
Ninety percent of patients reported their symptoms were controlled with adherence to diet after treatment with volanesorsen vs. 55% at baseline, according to the researchers.
Median number of symptoms decreased from 6.5 at baseline to 3.5 after volanesorsen treatment (improvement, 34%; P < .05), Arca and colleagues wrote, noting that 41% of patients experienced more than 10 symptoms at baseline, which decreased to 14%. Reduction was seen across physical, emotional and cognitive domains.
Pancreatic pain was reported in eight patients at baseline and one after treatment, steatorrhea was reported in five patients at baseline and none after treatment, physical weakness/asthenia was reported in six patients at baseline and two after treatment, fatigue was reported in 10 patients at baseline and four after treatment, and back pain was reported in eight patients at baseline and four after treatment (P < .05 for all), the researchers wrote.
Emotional symptoms such as constant worry about having a pain attack at any time, feeling out of control and anger/frustration at having the disease were also reduced after treatment (P < .05 for all), they wrote.
According to the survey, after treatment with volanesorsen, patients said overall management of symptoms improved, interference with work and school decreased and negative effects on personal, social and professional life were reduced.
“For people living with FCS, it impacts every part of daily life, often making it difficult to work, care for other family members or even join in life events with family and friends. Studies like ... Re-FOCUS can enable patients and caregivers around the world to better understand and communicate about the devastating effects of this rare disease,” Lindsey Sutton, co-president of the FCS Foundation, said in a press release. – by Erik Swain
Disclosures: The study was funded by Akcea Therapeutics. Arca reports he received grants/honoraria from Aegerion, Akcea, Amgen, Ionis Pharmaceuticals, Merck Sharpe & Dohme, Mylan, Pfizer and Sanofi. Please see the study for the other authors’ relevant financial disclosures.
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